Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Fairview University Medical Center

Status: Unknown

Conditions: Globoid Cell Leukodystrophy; Niemann-Pick Disease; Adrenoleukodystrophy; Metachromatic Leukodystrophy; Mucopolysaccharidosis VI; Mucopolysaccharidosis I; Mannosidosis; Fucosidosis; Wolman Disease; Gaucher's Disease; Pulmonary Complications; I Cell Disease

Study type: Observational

Funder types: Other

Identifiers

NCT00005900

UMN-MT-1999-18

UMN-MT-9818

199/15111

Details and patient eligibility

About

OBJECTIVES: I. Evaluate bronchoalveolar lavage fluid and serum obtained from pediatric patients with storage disorders prior to allogeneic hematopoietic stem cell transplantation (HSCT) for the presence of proinflammatory cytokines and for the production of nitric oxide by alveolar macrophages to identify possible risk factors for pulmonary complications.

II. Investigate the underlying mechanism for the development of significant pulmonary complications in these patients during HSCT.

III. Evaluate bronchoalveolar lavage fluid and serum obtained from these same patients at the time a pulmonary complication develops post-HSCT, or at 60 days post-HSCT if there has been no pulmonary complications.

Full description

PROTOCOL OUTLINE:

Patients undergo bronchoscopy with bronchoalveolar lavage (BAL) prior to allogeneic hematopoietic stem cell transplantation (HSCT). ELISA assays for cytokines are performed. Patients are followed post-HSCT for the development of transplant related pulmonary complications. A repeat bronchoscopy with BAL is performed at the time pulmonary complications develop or at day 60 post-HSCT if no complications develop. Cytokine assays are repeated.

Sex: All

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

• Diagnosis of an inborn error of metabolism eligible for allogeneic hematopoietic stem cell transplantation on protocol UMN-MT-1995-01